Safety Evaluation of MSC-based Therapy for Liver Cihcrosis Treatment

Phase 1 Clinical Trial: Evaluation of the Safety and Preliminary Efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell Extracellular Vesicle Therapy in the Treatment of Liver Cirrhosis

This study Phase 1 clinical trial aimed to evaluate the safety and preliminary efficacy of intravenously administered extracellular vesicles derived from umbilical cord mesenchymal stem cells (UC-MSC-EVs; VinEV-3) in patients with liver cirrhosis. The trial uses a rolling six dose-escalation design, enrolling up to 12 adult patients (18-75 years) with Child-Pugh scores of 7-12.

The results of this study are expected to provide initial clinical evidence supporting the safety and potential therapeutic role of UC-MSC-EVs as a novel cell-free treatment approach for liver cirrhosis.

Study Overview

• Status: Not yet recruiting
• Conditions: Liver Cirrhosis
• Intervention / Treatment: Biological: Umbilical cord mesenchymal stem cell-derived extracellular vesicles

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Thanh Liem Nguyen; Phone Number: (+84) 98 656 50 15; Email: v.liemnt@vinmec.com
• Study Contact Backup: Name: Van T. Hoang; Phone Number: +84 93 644 94 81

Study Locations

• Vietnam
  • Hanoi, Vietnam, 100000
    • Vinmec Research Institute of Stem Cell and Gene Technology
    • Contact: Thanh Liem Nguyen; Phone Number: +84 4 3974 3556; Email: v.liemnt@vinmec.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Liver cirrhosis due to alcohol-related liver disease or chronic hepatitis B or C
• Child-Pugh score 7-12
• Alcohol-related cirrhosis: abstinent from alcohol ≥ 3 months
• HBV/HCV-related cirrhosis: viral disease controlled according to standard clinical criteria
• Written informed consent provided

Exclusion Criteria:

• Significant renal dysfunction or coagulation abnormalities
• Liver cirrhosis of unknown etiology
• Current or suspected hepatocellular carcinoma or history of malignancy
• Portal vein thrombosis
• Pregnancy, breastfeeding, or inadequate contraception
• Severe renal, respiratory, cardiovascular, infectious, autoimmune, metabolic, or neurological disorders that may interfere with study participation
• Refractory ascites at screening
• Use of known hepatotoxic medications within a clinically relevant period
• Coinfection with HIV, tuberculosis, or other causes of chronic liver disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Treatment Regimen; Participants in this experimental arm will receive intravenous VinEV-3. Premedication with Dimedrol will be administered prior to infusion. VinEV-3 will be diluted in 0.9% sodium chloride to a total volume of 100 mL and infused over approximately 60 minutes. A dose-escalation design will be applied with safety-based dose adjustment. Participants will receive three infusions administered at 30 ± 5 day intervals, while continuing standard-of-care treatment. Patients will be followed for safety and clinical outcomes at 3, 6, and 9 months after the first infusion

Biological: Umbilical cord mesenchymal stem cell-derived extracellular vesicles; Dimedrol 20 mg will be administered intravenously 15-30 minutes prior to EV infusion. VinEV-3 will be administered at a starting dose of 2 × 10¹⁰ EV particles/kg, with dose escalation to 4 × 10¹⁰ EV particles/kg in the absence of dose-limiting toxicity or dose reduction to 1 × 10¹⁰ EV particles/kg if dose-limiting toxicity occurs. Three infusions will be given at 30 ± 5 day intervals, 3 times, with safety follow-up through 9 months after the first infusion; Other Names: UC-MSC-EV; VinEV-3

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency and Severity of Adverse Events and Serious Adverse Events	Incidence, type, and severity of adverse events (AEs) and serious adverse events (SAEs) graded according to CTCAE v5.0 and dose-limiting toxicities (DLTs) when administered VinEV-3 at escalating dose levels.	9 months from first infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Blood serum biochemical analysis	Assessment of changes in serum albumin (ALB), AST, ALT, GGT, ALP levels	Baseline, day 1, day 30, day 31, day 60, day 61, day 90, day 180, day 270
Child-Pugh score	The Child-Pugh score is used to assess the prognosis of chronic liver disease, mainly cirrhosis. It consists of five clinical measures: total bilirubin, serum albumin, prothrombin time (INR), ascites, and hepatic encephalopathy. The total score ranges from 5 to 15, where higher scores indicate worse hepatic impairment (Class A: 5-6 points, Class B: 7-9 points, and Class C: 10-15 points).	Baseline, day 30, day 60, day 90, day 180, day 270
MELD score	The Model for End-Stage Liver Disease (MELD) is a scoring system used to estimate the severity of chronic liver disease. The score is calculated using a formula that includes serum bilirubin, serum creatinine, and the international normalized ratio (INR). MELD scores range from 6 to 40, where a higher score indicates a more severe disease state and a higher risk of mortality	Baseline, day 30, day 60, day 90, day 180, day 270
Change in quality of life	Quality of life will be assessed using the Short Form-36 (SF-36) Health Survey. The survey consists of 36 questions covering 8 health domains. Each domain is scored on a scale of 0 to 100, where higher scores indicate a better quality of life and better health status.	Baseline, day 90, day 180, day 270

Collaborators and Investigators

• Sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
• Investigators: Principal Investigator: Thanh Liem Nguyen, Vinmec Research Institute of Stem Cell and Gene Technology Hanoi, Vietnam 100000

Study record dates

Study Major Dates

• Study Start (Estimated): March 15, 2026
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: February 5, 2026
• First Submitted That Met QC Criteria: March 13, 2026
• First Posted (Actual): March 16, 2026

Study Record Updates

• Last Update Posted (Actual): March 16, 2026
• Last Update Submitted That Met QC Criteria: March 13, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Digestive System Diseases; Liver Diseases; Fibrosis; Pathological Conditions, Signs and Symptoms; Liver Cirrhosis
• Other Study ID Numbers: ISC.25.01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No